Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (September 2018)
24 slides from Neuren Pharmaceuticals's investor presentation, Sep 6, 2018
Slide 1: Investor Presentation
For personal use only Investor Presentation 6 September 2018 1
Slide 2: Forward looking statements
Forward looking statements For personal use only This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations reflected in the forward looking statements are reasonable at this time, Neuren can give no assurance that these expe
Slide 3: Neuren snapshot
Neuren snapshot For personal use only New therapies for neurological conditions with high unmet need Significant commercial opportunities with no approved drugs Regulatory incentives – Orphan Drug, Fast Track, Priority Review Strong support from advocacy groups and leading physicians Trofi
Slide 4: Normal biology of IGF-1 in the brain
Normal biology of IGF-1 in the brain For personal use only Produce essential growth factor Reversible binding IGF-1 IGF-1 regulates bioavailability IGF Binding Protein-3 Enzyme cleavage Regulates binding Truncated IGF-1 Enzyme cleavage GPE cGP Activates PI3K–Akt–mTOR and Ras–MAPK- ERK signalling pat
Slide 5: Key features of ACADIA partnership
Key features of ACADIA partnership For personal use only ACADIA granted exclusive license for trofinetide in North America Neuren retains all rights outside North America Free use of all data and regulatory information generated by ACADIA in the US for registration outside North America Neur
Slide 6: Key features of ACADIA partnership
Key features of ACADIA partnership For personal use only ACADIA team has proven record developing and commercializing therapies for CNS disorders in the US that have no approved therapies and high unmet need ACADIA will fund all development costs for trofinetide in North America (including ~US$5
Slide 7: Rett syndrome development program
Rett syndrome development program For personal use only Statistically significant and clinically meaningful improvement demonstrated in pediatric Phase 2 clinical trial Phase 3 program with single trial agreed at End of Phase 2 Meeting with FDA Division of Neurology Products ACADIA to commence
Slide 8: Trofinetide commercial exclusivity
Trofinetide commercial exclusivity For personal use only Issued composition of matter patents owned by Neuren US and Europe – expire 2022, potential to extend to 2027 Exclusivity periods from orphan drug designation US – 7 years from marketing authorization, potentially plus 6 months if appr
Slide 9: Advancing NNZ-2591
Advancing NNZ-2591 For personal use only Demonstrated efficacy in pre-clinical models of Parkinson’s disease, stroke, traumatic brain injury, peripheral neuropathy, Fragile X syndrome, memory impairment and multiple sclerosis Funds now available to accelerate CMC and pre-clinical development req
Slide 10: Neuren stock information
Neuren stock information (ASX: NEU) For personal use only Quoted shares: 100.2 million 52 week range: $1.04 - $3.56 2.5 million loan funded shares at exercise prices $1.62 - $1.84 (Fully diluted shares: 102.7 million) Langley Management, Walker, 18% 10% Institutions, 12% Retail, 60% 10
Slide 11: Neuren's near-term priorities
Neuren’s near-term priorities For personal use only Actively support ACADIA in development of trofinetide for North America Complete second non-clinical chronic toxicology study Assist with preparations for Rett syndrome Phase 3 trial: Manufacture of drug supplies Finalise protocol, sites
Slide 12: Trofinetide in Rett syndrome and Fragile X syndrome
For personal use only Trofinetide in Rett syndrome and Fragile X syndrome 12
Slide 13: About Rett syndrome
About Rett syndrome For personal use only Seriously debilitating and life-threatening neurological disorder, with no approved medicines Caused by non-inherited mutation on the X chromosome – estimated 1 in 10,000 to 15,000 live female births in all racial and ethnic groups After apparently nor
Slide 14: Results of Phase 2 trial in girls aged 5 to 15
Results of Phase 2 trial in girls aged 5 to 15 For personal use only Double-blind, placebo-controlled Phase 2 trial of treatment for 6 weeks in girls with Rett syndrome, aged 5 to 15 years Conducted at 12 US hospitals, led by world-leading clinicians in Rett syndrome and supported by rettsyndrom
Slide 15: RSBQ – 200mg/kg versus placebo
RSBQ – 200mg/kg versus placebo For personal use only Mean improvement after 6 weeks of treatment Time course of Improvement A decrease on the y-axis indicates clinical improvement Mean improvements for 200mg/kg and placebo were, respectively, 16% and 6% of the treatment baseline Caregiver rate
Slide 16: CGI-I – 200mg/kg versus placebo
CGI-I – 200mg/kg versus placebo For personal use only Mean improvement after 6 weeks of treatment Time course of Improvement A decrease on the y-axis indicates greater clinical improvement Clinician rates how much the subject’s overall illness has improved or worsened, relative to baseline, with
Slide 17: Views of leading physicians and rettsyndrome.org
Views of leading physicians and rettsyndrome.org For personal use only Walter Kaufmann, MD: “The outcome of this trial is very encouraging. Safety, the primary goal, was achieved. As important and with broad implications, there was a clear clinical improvement covering several common symptoms in Ret
Slide 18: Impact in mouse model of Rett syndrome
Impact in mouse model of Rett syndrome For personal use only In “Partial reversal of Rett syndrome like symptoms in MeCP2 mutant mice” (doi:10.1073/pnas.0812394106), Tropea et al reported that in the MeCP2 knockout mouse, introducing GPE: • Extended life span, improved locomotor function, ameliorate
Slide 19: Impaired brain biology and trofinetide intervention
Impaired brain biology and trofinetide intervention For personal use only In Rett syndrome, synapses are too few and underdeveloped, due to: Insufficient formation of new synapses by neurons Exaggerated maintenance of existing synapses by overactive microglia A mutation in the MECP2 gene cau
Slide 20: Trofinetide in Fragile X syndrome
Trofinetide in Fragile X syndrome For personal use only Inherited X chromosome mutation – full mutation causes Fragile X syndrome 1 / 4,000 males and 1 / 6,000 females estimated to have full mutation More severe in males, ~50% of females have some features of the syndrome The most common inh
Slide 21: Completed Phase 2 trial in adolescents and adults
Completed Phase 2 trial in adolescents and adults For personal use only Double-blind, placebo-controlled Phase 2 trial of 28 days of treatment with two dose levels 70 subjects aged 12 to 45 years received treatment, with 68 subjects completing the trial Both dose levels were well tolerated and
Slide 22: Core efficacy measures that met target
Core efficacy measures that met target For personal use only Fragile X Syndrome Rating Scale Fragile X Domain Specific Concerns Aberrant Behavior Checklist (ABC) Total Score Analysis of mean clinical responses at end of treatment for each treatment group A negative value on the y-axis indicates
Slide 23: Impact in mouse model of Fragile X syndrome
Impact in mouse model of Fragile X syndrome In the fmr1 knockout mouse model, trofinetide normalised mutant For personal use only mice, but had no effect on wild type mice: • Corrected learning and memory deficits, hyperactivity and social behaviour • Reduced dendritic spine density • Normalised ove
Slide 24: Impaired brain biology and trofinetide intervention
Impaired brain biology and trofinetide intervention For personal use only Mutations in the fmr1 gene cause a deficit in production of the Fragile X Mental Retardation Protein (FMRP), which leads to too many and immature dendritic spines and abnormal synaptic transmission: Impaired functioning of
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